Browsing Tag
Duchenne muscular dystrophy
31 posts
Atossa Therapeutics (NASDAQ: ATOS) expands (Z)-endoxifen to DMD: Will investors buy the thesis?
Atossa Therapeutics eyes new ground in DMD care with (Z)-endoxifen. Find out how this small molecule could reshape treatment for carriers and boys alike.
Why the FDA’s warning on Elevidys matters for Duchenne muscular dystrophy treatment
Sarepta’s Elevidys gene therapy now carries an FDA boxed warning. Find out what it means for Duchenne patients, investors, and gene therapy safety.
Sarepta Therapeutics (NASDAQ: SRPT) hit by FDA boxed warning as ELEVIDYS loses non‑ambulatory DMD indication
Sarepta’s ELEVIDYS gene therapy label now includes a boxed liver warning and removes non-ambulatory DMD use. Explore the safety updates and investor outlook.
What makes gene-editing tools like CRISPR 3.0 the next frontier in precision medicine?
Delve into the next generation of gene-editing tools, such as prime editing, and see how they may revolutionize precision medicine – read more now!
Sarepta Therapeutics awards 25 Route 79 scholarships in 2025–2026, but what makes this Duchenne program unique?
Sarepta Therapeutics awarded 25 Route 79 scholarships for 2025–2026 to support students impacted by Duchenne. See how this initiative strengthens community ties.
Capricor Therapeutics (NASDAQ: CAPR) reports sustained efficacy and safety in 4-year DMD trial of Deramiocel
Capricor Therapeutics reports 4-year HOPE-2 data for Deramiocel in Duchenne, showing cardiac stability and slowed muscle decline. Read how it may shape future FDA decisions.
Sarepta’s gene therapy marches on: UK clears continued dosing in Duchenne Phase 3 trial
Find out how Sarepta Therapeutics is advancing Duchenne gene therapy with UK backing for its ENVISION trial of ELEVIDYS in non-ambulatory patients.
Breakthrough data from Vatiquinone trials propels PTC Therapeutics stock to 52-week high
PTC Therapeutics Inc. (NASDAQ: PTCT) is experiencing significant momentum within the biotechnology sector following a surge in its…
Capricor Therapeutics announces promising long-term results for deramiocel deramiocel in Duchenne muscular dystrophy
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company at the forefront of developing cell and exosome-based therapies, has shared…
FDA approves Italfarmaco’s Duvyzat for duchenne muscular dystrophy treatment
In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S.…